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Dr Kieran Breen is currently the European Parkinson’s Disease Association representative on the European Medicines Agency Committee for Advanced Therapies, which oversees new product application analysis and the development of product classification guidelines for advanced therapies. He has a PhD in neuroscience and was the director of research and innovation at Parkinson’s UK for nine years.

The emerging category of medicines called advanced therapeutic medicinal products may have great potential to develop new treatments, and potentially cures, for Parkinson’s

Dr Kieran Breen

The future generation of advanced therapies for Parkinson’s

Special reports

Author: Dr Kieran BreenPublished: 22 October 2015

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In this special report, Dr Kieran Breen of the European Medicines Agency Committee for Advanced Therapies, explores some of the most exciting areas of research for potential Parkinson’s treatments and cures – including stem cell-based therapy and gene therapy

The development of advanced therapies is an extremely exciting field, which offers renewed hope for the treatment of conditions such as Parkinson’s. Although only a small number have been approved for use in the clinic so far, the number that are in early stage clinical trials suggests that this is a really promising area.

While the current Parkinson’s therapies address the symptoms of the condition, there are no treatments available that will modify its progression. However, the emerging category of medicines called advanced therapeutic medicinal products (ATMPs) may have great potential to develop new treatments, and potentially cures, for Parkinson’s.

The first category, “somatic cell medicinal products”, describes cells which have been obtained from tissue, such as bone marrow, and have then been manipulated to generate a cell which has a different function. These can then be administered either as a treatment or as a diagnostic tool.

Example: Holoclar is used to treat certain rare forms of blindness

2. “Gene therapy”

The second is called “gene therapy” where specific genes are introduced into the body, either alone or within transplanted cells. The primary aim is to overcome genetic mutations associated with specific conditions and which may prevent the proteins, which are derived from the genes, from working properly.

Example: Glybera is used to treat digestive disorders

3. “Tissue-engineered products”

The third class is “tissue-engineered products”. These are cells or tissues that are used to stimulate the regeneration or repair damaged tissue within the body. However, the cells or tissues will not have the same function in the recipient as in the donor. Otherwise, it would be classed as a tissue transplant and this is not considered to be a medicine in legal terms.

Example: ChondroCelect is used to repair damage to the cartilage in the knee

The biggest potential outcome is within stem cell-based therapy. The initial stem cell-based product, Holoclar, was approved for the treatment of a rare eye condition that can result in blindness. This demonstrates that, at least in principle, stem cells can be manipulated to replace cells that may be damaged. In the future, it may be possible to replace the nerve cells affected in Parkinson’s in a similar way.

The second significant advance is in the area of gene therapy where a single injection of a gene into a nerve cell could give rise to a continuous production of the associated protein. One therapy where this may be useful is growth factors. The GDNF protein, which is currently being tested in a clinical trial, stimulates dying nerve cells to regrow and may reverse the progression of Parkinson’s. However, because of its size, it has to be regularly injected directly into the brain. An alternative approach could be to give a single injection of the gene and thus allow the nerve cells to produce their own GDNF.

While these approaches may be a number of years off, and there is no guarantee that they would be effective, the existing evidence suggests that ATMPs may have real promise for the future treatment of Parkinson’s.

Share this story

Dr Kieran Breen is currently the European Parkinson’s Disease Association representative on the European Medicines Agency Committee for Advanced Therapies, which oversees new product application analysis and the development of product classification guidelines for advanced therapies. He has a PhD in neuroscience and was the director of research and innovation at Parkinson’s UK for nine years.

The emerging category of medicines called advanced therapeutic medicinal products may have great potential to develop new treatments, and potentially cures, for Parkinson’s

IN THE NEWS

Carefully selected news stories from the international Parkinson's community.

3 weeks ago

Excess calcium in brain could cause Parkinson’s

Researchers at the University of Cambridge, UK, have discovered that excess levels of calcium in brain cells may lead to the formation of the toxic clusters that signify Parkinson’s disease. The findings, reported in the journal ‘Nature Communications’, show that calcium can influence the interaction between small membranous structures inside nerve endings, which are important for neuronal signaling in the brain, and alpha-synuclein – the protein associated with Parkinson’s disease. Dr Janin Lautenschläger, the paper’s first author, said: “This is the first time we’ve seen that calcium influences the way alpha-synuclein interacts with synaptic vesicles. We think that alpha-synuclein is almost like a calcium sensor. In the presence of calcium, it changes its structure and how it interacts with its environment, which is likely very important for its normal function.”

Jewish people with Crohn’s disease more likely to carry LRRK2 gene mutation

A scientific study has concluded that there may be a link between Parkinson’s and Crohn’s disease within the Ashkenazi Jewish community. The study’s findings, which were published in the journal ‘Science Translational Medicine’, has found that members of the population with Crohn’s disease are more likely to carry the LRRK2 mutation which is a significant cause of Parkinson’s. Lead researcher Dr Inga Peter, professor of genetics and genomic sciences at the Icahn School of Medicine, New York, US, said: “Crohn’s disease is a complex disorder with multiple genes and environmental factors involved, which disproportionately affects individuals of Ashkenazi Jewish ancestry. “The presence of shared LRRK2 mutations in patients with Crohn’s disease and Parkinson’s disease provides refined insight into disease mechanisms and may have major implications for the treatment of these two seemingly unrelated diseases.”

Could caffeine in the blood help diagnose Parkinson’s?

Blood caffeine levels could be promising diagnostic biomarkers for early-stage Parkinson’s, Japanese researchers reported in the journal ‘Neurology’ earlier this month. The study found that people with Parkinson’s had lower levels of caffeine and caffeine metabolites in their blood than people without the disease, at the same consumption rate. Caffeine concentrations also were decreased in Parkinson’s patients with motor fluctuations than in those without Parkinson’s. However, patients in more severe disease stages did not have lower caffeine levels. The study’s authors, Dr David Munoz, University of Toronto, and Dr Shinsuke Fujioka, Fukuoka University, suggested that the “decrease in caffeine metabolites occurs from the earliest stages of Parkinson’s.” They added: “If a future study were to demonstrate similar decreases in caffeine in untreated patients with Parkinson’s […] the implications of the current study would take enormous importance.”